Long-term safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis type II: 2-year results from a phase 2/3 extension study
Title:
Long-term safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis type II: 2-year results from a phase 2/3 extension study
Author:
Muenzer, Joseph Burton, Barbara K. Harmatz, Paul Gutiérrez-Solana, Luis González Ruiz-Garcia, Matilde Jones, Simon A. Guffon, Nathalie Inbar-Feigenberg, Michal Bratkovic, Drago Li, Xiaoxi Yee, Karen S. Alexanderian, David